OBJECTIVES: Currently, China has initially established a multi-payer reimbursement system (MPRS) with basic medical insurance (BMI) as the main body, medical aid as the fallback, and commercial health insurance in parallel. The study aims to discuss current situation of rare disease patients’ affordability in current MPRS in China and possible improvement measures.

METHODS: Based on the BMI reimbursement policies of 250 cities in China in 2023, the World Health Organization/Health Action International (WHO/HAI) standardized survey methodology was used to evaluate the affordability of 15 rare disease drugs for the treatment of nine rare diseases, including multiple sclerosis, Fabray disease, and Gaucher disease.

RESULTS: The affordability of five low-value drugs for Parkinson's disease (young and early onset), systemic sclerosis and multiple sclerosis was good (requiring 0.03-0.80 years of payment) with BMI reimbursement only, while four high-value drugs for hemophilia, Niemann-Pick disease and Fabray's disease required more than 1 year of payment, and only Magestat was not affordable after secondary reimbursement by the Customized City Commercial Medical Insurance (CCCMI), requiring 1.97 years of payment. The six high-value rare disease drugs that are covered only through the CCCMI are the least affordable, requiring more than 15 years of payment.

CONCLUSIONS: A MPRS is essential to improve patients' access to high-value rare disease drugs, therefore it is necessary to optimize MPRS and improve policy convergence from CCCMI to BMI. In addition, for high-value rare disease drugs that are difficult to be included in BMI, or with limited reimbursement, option of an independent rare disease fund could be explored to improve patients' affordability.